Huntington’s disease is a severe progressive neurological disorder caused by a CAG-repeat expansion in the HTT gene. A small molecule shows therapeutic potential by inducing contraction of these expanded CAG repeats in cell and mouse models of the disease.
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M.D.F. has no competing interests. S.J.T. is on scientific advisory boards for Triplet Therapeutics and LoQus therapeutics and has laboratory funding from Vertex pharmaceuticals.
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Flower, M.D., Tabrizi, S.J. A small molecule kicks repeat expansion into reverse. Nat Genet 52, 136–137 (2020). https://doi.org/10.1038/s41588-020-0577-6